HOUSE PASSES MATSUI’S RARE ACT

WASHINGTON, D.C. - Today, Congresswoman Doris Matsui (CA-07), co-chair of the Rare Disease Congressional Caucus, released the following statement after the House passed the Give Kids a Chance Act, a package of rare disease legislation that included her Retaining Access and Restoring Exclusivity (RARE) Act. The RARE Act would ensure that promising options for rare disease patients with no approved therapies are able to come to market without being blocked by other pharmaceutical companies.

"With less than five percent of rare diseases having FDA-approved therapies, we need all the help we can get to ensure rare disease patients can access the cutting-edge care they need," said Congresswoman Matsui. "The RARE Act will prevent pharmaceutical companies from abusing their orphan drug status to keep other innovative drugs from coming to market. Every rare disease patient deserves a fighting shot. Their access to medicine shouldn't be sacrificed for drug companies' bottom line."

Specifically, the RARE Act clarifies FDA's longstanding interpretation of the Orphan Drug Act to ensure that the scope of the orphan drug exclusivity applies only to the same approved use or indication within such rare disease or condition, instead of the same disease or condition. This will give FDA the necessary authority to approve the same drug from different manufacturers if they aim to serve different patient populations.

Full text of the RARE Act is available HERE.

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